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Study of Oral MC-1 for the Treatment of Patients with PNPO Deficiency
2025-522346-46-00Phase 3Authorised
- Sponsor
- Medicure Inc.
- Start
- Nov 2025
- Modality
- Orthosteric Ligands → Classical Binding Small Molecules → Small Molecules, Metabolically Activated Prodrugs → Prodrugs/Conditional Activator Small Molecules → Small Molecules
- Countries
- 1
01
Overview
Phase 3, single-arm trial of pyridoxal-5'-phosphate in pyridoxamine 5'-phosphate oxidase deficiency, sponsored by Medicure Inc. Primary endpoint: Incidence of death at 12 months. Planned start Nov 2025; status: authorised.
02
Drugs
pyridoxal-5'-phosphate
03
Conditions
pyridoxamine 5'-phosphate oxidase deficiency
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